LWPES: Early Growth Hormone Therapy Changes Prader-Willi Course

Action Points

Explain to interested patients that this study showed that early treatment with human growth hormone may improve the strength and condition of children with Prader-Willi syndrome.

Note that this study was published as an abstract and presented at a conference. These data and conclusions should be considered preliminary until published in a peer-reviewed journal.

VANCOUVER -- Early identification and treatment of children with Prader-Willi syndrome (PWS) might be able to change the course of a growth disorder characterized by obesity and an urge to keep eating, researchers said here.

By treating these children with recombinant human growth hormone (hGH) from infancy until they were 6 to 7 years old, researchers were able to improve their physiques and physical strength compared with untreated children, according to Aaron Carrel, MD, of the University of Wisconsin in Madison.

He reported his group's findings from a randomized study of 48 children with Prader-Willi syndrome at the meeting of the Lawson Wilkins Pediatric Endocrine Society, held in conjunction with the Pediatric Academic Societies' annual meeting here.

One group of 21 children had been treated with hGH for about six years after their first birthday. The other group of 27 children of about the same age had not yet been treated with hGH. Researchers compared the physical development of the two groups.

Compared with nontreated PWS children, those children treated with hGH:

Carrel said a hallmark of PWS was the accumulation of fat, without a corresponding accumulation of muscle. Fat accumulation by itself, he noted, affects growth hormone production and the youngster's overall well-being.

He said that because studies have shown that growth hormone improves outcomes in PWS, ethical concerns prevent conducting a randomized, placebo-controlled trial to see how treatment impacts the natural history of the disease.

But the results he observed by comparing the two cohorts indicate that "long-term human growth hormone therapy favorably alters the natural history of Prader-Willi syndrome to an extent that exceeds risks and justifies consideration for initiation during infancy."

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